Ignite Creation Date:
2025-12-25 @ 2:11 AM
Ignite Modification Date:
2025-12-30 @ 9:52 AM
Study NCT ID:
NCT00104260
Status:
COMPLETED
Last Update Posted:
2007-04-09
First Post:
2005-02-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010661', 'term': 'Phenylketonurias'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C003402', 'term': 'sapropterin'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'count': 700}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2004-12'}, 'statusVerifiedDate': '2007-04', 'completionDateStruct': {'date': '2005-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2007-04-05', 'studyFirstSubmitDate': '2005-02-24', 'studyFirstSubmitQcDate': '2005-02-24', 'lastUpdatePostDateStruct': {'date': '2007-04-09', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2005-02-25', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluate the degree and frequency of response to Phenoptin™, as demonstrated by a reduction in blood Phe level among subjects with PKU who have elevated Phe levels'}], 'secondaryOutcomes': [{'measure': 'Evaluate the safety of Phenoptin™ treatment in this subject population, and identify individuals in this subject population who respond to Phenoptin™ treatment with a reduction in blood Phe level'}]}, 'conditionsModule': {'conditions': ['Phenylketonurias']}, 'descriptionModule': {'briefSummary': 'The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects with phenylketonuria (PKU) who have elevated Phe levels. A secondary objective of this study is to evaluate the safety of Phenoptin™ treatment in this subject population, and identify individuals in this subject population who respond to Phenoptin™ treatment with a reduction in blood Phe level.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '8 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age \\>/= 8 years\n* Blood Phe level \\>/= 450 umol/L at screening\n* Clinical diagnosis of PKU with hyperphenylalaninemia documented by past medical history of at least one blood Phe measurement \\>/= 360 umol/L (6 mg/dL)\n* Willing and able to provide written informed consent or, in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained\n* Negative urine pregnancy test at screening (non-sterile females of child-bearing potential only)\n* Male and Female subjects of childbearing potential childbearing potential (if sexually active and non-sterile) must be using acceptable birth control measures, as determined by the investigator, and willing to continue to use acceptable birth control measures while participating in the study\n* Willing and able to comply with study procedures\n* Willing to continue current diet unchanged while participating in the study\n\nExclusion Criteria:\n\n* Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable\n* Use of any investigational agent within 30 days prior to screening, or requirement for any investigational agent or vaccine prior to completion of all scheduled study assessments\n* Pregnant or breastfeeding, or considering pregnancy\n* ALT \\> 5 times the upper limit of normal (i.e., Grade 3 or higher based on World Health Organization Toxicity Criteria) at screening\n* Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes, or organ transplantation)\n* Serious neuropsychiatric illness (e.g., major depression) not currently under medical control\n* Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)\n* Concurrent use of levodopa\n* Clinical diagnosis of primary BH4 deficiency'}, 'identificationModule': {'nctId': 'NCT00104260', 'briefTitle': 'Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels', 'orgStudyIdInfo': {'id': 'PKU-001'}}, 'armsInterventionsModule': {'interventions': [{'name': 'sapropterin dihydrochloride', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'locations': [{'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'city': 'Oakland', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'city': 'New York', 'state': 'New York', 'country': 'United States', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}}}}